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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02344004
Registration number
NCT02344004
Ethics application status
Date submitted
9/01/2015
Date registered
22/01/2015
Date last updated
7/05/2020
Titles & IDs
Public title
Study to Evaluate Efficacy of LAI When Added to Multi-drug Regimen Compared to Multi-drug Regimen Alone
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Scientific title
A Randomized, Open-label, Multicenter Study of Liposomal Amikacin for Inhalation (LAI) in Adult Patients With Nontuberculous Mycobacterial (NTM) Lung Infection Caused by Mycobacterium Avium Complex (MAC) That Are Refractory to Treatment
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Secondary ID [1]
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2014-005010-31
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Secondary ID [2]
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INS-212
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Universal Trial Number (UTN)
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Trial acronym
CONVERT
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Mycobacterium Infections, Nontuberculous
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Condition category
Condition code
Infection
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Studies of infection and infectious agents
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Infection
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Other infectious diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - LAI (Liposomal Amikacin for Inhalation) 590 mg
No Intervention: Multi-drug Regimen - Participants received their already prescribed anti-mycobacterial regimen (based on the 2007 American Thoracic Society/Infectious Diseases Society of America [ATS/IDSA] Guidelines)
Experimental: LAI + Multi-drug Regimen - Participants received LAI 590 mg QD in addition to their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines)
LAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes
Treatment: Drugs: LAI (Liposomal Amikacin for Inhalation) 590 mg
LAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of Participants Achieving Culture Conversion by Month 6 in the Liposomal Amikacin for Inhalation (LAI) + Multi-drug Regimen (MDR) Arm Compared to the MDR Alone Arm
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Assessment method [1]
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Sputum specimens were collected at Screening (Visit 1), Baseline (Visit 2), and at Visits 3 (Month 1) through 8 (Month 6). A negative culture result reflected a negative culture result for all sputum samples collected at each visit. Participants met the primary endpoint of culture conversion by Month 6 if they had 3 consecutive monthly MAC-negative sputum cultures during the first 6 months of the study. A participant needed to achieve the first of 3 consecutive negative sputum cultures (that defined culture conversion) by Month 4 in order to meet the primary endpoint by Month 6. Each participant in the intent to treat (ITT) population (ie, all randomized participants) was classified as either a converter or non-converter by Month 6.
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Timepoint [1]
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by Month 6
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Primary outcome [2]
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Number of Participants Achieving Durable Culture Conversion Through 3 Months Off Treatment in the LAI + MDR Arm Compared to the MDR Arm Alone
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Assessment method [2]
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Sputum specimens were collected at screening, baseline (Day 1), during treatment, and at Months 1, 3, 6, and 12 months off treatment. Culture conversion with durability was defined as achieving culture conversion by Month 6 and then having no more than 2 consecutive broth positive cultures and no Agar positive culture up to 3 months off treatment. Converters with missing broth or Agar sputum culture result after Month 6 up to 3 months off treatment were considered as not achieving culture conversion with durability except those participants who are unable to produce sputum despite reasonable efforts, as reported by source documentation. Participants who had relapse/recurrence, had "rescue" medication and/or died before reaching 3 months off treatment were considered as not achieving culture conversion with durability.
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Timepoint [2]
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up to Month 19
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Secondary outcome [1]
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Change From Baseline (Day 1) to Month 6 in the Six-Minute Walk Test (6MWT) Distance in the LAI + MDR Arm Compared to the MDR Alone Arm
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Assessment method [1]
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A 6-minute walk assessment of exertional capability was performed at Baseline (Day 1) and at Month 6. The standardized protocol based on the ATS guidelines was used. The 6MWT was conducted by a site member who was blinded to the participant's open-label treatment assignment. The analysis of the change from Baseline (Day 1) to Month 6 in the 6MWT distance was performed after the last participant completed Month 6 and his/her 6MWT distance data were available.
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Timepoint [1]
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at Month 6
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Secondary outcome [2]
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Time to Culture Conversion by Month 6 in the LAI + MDR Arm Compared to the MDR Alone Arm
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Assessment method [2]
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The time to culture conversion was defined by the date of the first of at least 3 consecutive monthly culture specimens that were Mycobacterium avium complex (MAC)-negative.
The 25th percentile time to conversion is the estimated time taken for 25% of participants to convert.
The 50th percentile time to conversion is the estimated time taken for 50% of participants to convert.
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Timepoint [2]
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by Month 6
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Secondary outcome [3]
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Number of Participants Achieving Sustained Culture Conversion at the End of Treatment (EOT) in the LAI + MDR Arm Compared to the MDR Arm Alone
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Assessment method [3]
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Sustained conversion was evaluated in participants who completed at least 12 months of treatment from the start of culture conversion. Sustained conversion was defined as conversion (3 consecutive negative monthly sputum samples) by Month 6 with no positive agar media culture or no more than 2 broth media cultures up to and including the time point. Participants who did not convert were considered non-sustained conversions.
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Timepoint [3]
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up to Month 16
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Secondary outcome [4]
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Change in 6-Minute Walk Test (6MWT) Distance at EOT in the LAI Arm Compared to a Multi-drug Regimen Alone
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Assessment method [4]
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A 6-minute walk assessment of exertional capability was performed at Baseline (Day 1) and up to EOT or Month 16. The standardized protocol based on the ATS guidelines was used. The 6MWT was conducted by a site member who was blinded to the participant's open-label treatment assignment.
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Timepoint [4]
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up to Month 16
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Secondary outcome [5]
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Change From Baseline (Day 1) at Month 6 in the St. George's Respiratory Questionnaire (SGRQ) Total Score
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Assessment method [5]
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The SGRQ was completed before administration of study drug at Baseline (Day 1) and Months 3, 6, 8, and 12, and at the EOT visit and the 3 months off treatment visit. The SGRQ is a self-administered questionnaire that has been validated in participants with airways disease, specifically in participants with bronchiectasis. The SGRQ assesses health-related quality of life in participants with chronic pulmonary disease by evaluating 3 health domains: symptoms (distress caused by respiratory symptoms); activity (effects of disturbances on mobility and physical activity); and impacts (the effect of disease on factors such as employment, personal control of one's health, and need for medication). A composite total score is derived as the sum of domain scores for symptoms, activity, and impact (0 = best possible score and 100 = worst possible score). A within patient reduction from baseline in score of 4 units is generally recognized as a clinically meaningful improvement in quality of life.
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Timepoint [5]
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at Month 6
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Eligibility
Key inclusion criteria
1. Be continually positive for MAC on sputum culture while adhering to a multi-drug
treatment regimen for a minimum duration of 6 months which is either ongoing or was
completed no more than 12 months before screening
2. Be diagnosed with MAC NTM lung infection with evidence of nodular bronchiectasis
and/or fibrocavitary disease by chest CT
3. Be willing to adhere to multi-drug treatment regimen during the course of the study
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
1. Patients with cystic fibrosis
2. Positive pregnancy test or lactation at screening. All women of child bearing
potential will be tested. Women not of childbearing potential are defined as
postmenopausal (i.e., amenorrheic for at least 1 year), or surgically or naturally
sterile.
3. Active pulmonary tuberculosis requiring treatment at screening
4. History of lung transplantation
5. Prior exposure to LAI (including clinical study).
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
27/05/2015
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
3/04/2019
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Sample size
Target
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Accrual to date
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Final
336
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Recruitment in Australia
Recruitment state(s)
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Insmed Incorporated
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
A study to evaluate the effectiveness of Liposomal Amikacin for Inhalation (LAI) 590 mg
administered once daily (QD) when added to multi-drug regimen (MDR) in participants with
Nontuberculous Mycobacterial (NTM) lung infection caused by Mycobacterium Avium Complex (MAC)
that were refractory to treatment.
Participants were randomized 2:1 to LAI 590 mg administered QD + MDR or MDR alone.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02344004
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Trial related presentations / publications
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Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed
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Contacts
Principal investigator
Name
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Gina Eagle, MD
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Address
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Insmed Incorporated
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02344004
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